Washington Organized medicine expects that a new initiative to create a national, patient-centered data infrastructure will not just expand upon and share patient data, but also benefit clinical registries that feed comparative effectiveness research.

On April 23, the Patient-Centered Outcomes Research Institute announced a series of funds to enlist the participation of physicians, patients and health care systems in building a national data network to advance research comparing the effectiveness of different treatment options on the same conditions. PCORI is a nonprofit, independent research organization that was created by the Affordable Care Act to fund such studies with the aim of enabling patients and clinicians to make better informed health care decisions.

Joe Selby, MD, MPH

The funds will promote a “network of networks” by creating two types of infrastructures that would improve the nation's ability to conduct this research. The new system actively would involve health care systems, clinicians and patients in the governance of these networks, while promoting data sharing across networks and collaboration with researchers, said Joe Selby, MD, MPH, executive director of PCORI. Dr. Selby and other members of the health care community discussed the new initiative at a Washington forum.

At least $56 million would be dedicated to eight new or existing “clinical data research networks” during the next 18 months. These networks would conduct randomized studies, incorporating data from large, defined populations in clinical practices. To qualify for funding, a network would need to demonstrate the ability to follow patients across care settings over time. Each network would consist of at least two health care systems and propose three pilots on specific conditions: diabetes or obesity patients; patients with another common disorder; and patients with a rare disorder.

An additional $12 million would support the growth and sustainability of 12 to 18 patient-powered research networks — patient groups who share various conditions and want to participate actively in this research. The purpose of these networks would be to test best practices in data collection and usage and encourage patients to become partners in research.

The expectation is that these two types of networks eventually would merge their efforts. For example, a clinical data research network might sponsor a clinical trial on a condition that a patient-powered network is focused on, resulting in the recruitment of interested patients. A steering committee with representation from all of the networks would help guide the integration.

“Actionable information” needed

Disease registries, medical groups and centers, patient organizations, integrated delivery systems and health plans have been invited to participate in, partner with or share data sources in this effort. For instance, a medical group could participate in a clinical data research network, combine its electronic health record data with those from another health care system, and then share the information with researchers who could study the data to compare treatment options for a certain health condition.

Janet Woodcock, MD

Initial reviews on PCORI's effort have been mostly positive. A good portion of medical care isn't evidence-based, said Janet Woodcock, MD, director of the Food and Drug Administration's Center for Drug Evaluation and Research. Not enough information has been available to show what the best course of treatment or intervention should be for any given person, she said. The FDA, which has been developing its own national electronic network, known as Sentinel, to improve the way it tracks the safety of medical devices, biologics and drugs, “is right in the middle of this, because we're engaged in overseeing the introduction of new drug treatments — and those have to be compared with what exists,” Dr. Woodcock said.

What the FDA and medical community need is “actionable information,” she said. “When we look a patient in the eye and say, 'You should take this. This is what we expect will happen. Here are your chances of getting a benefit,' we need solid evidence to back that up.” This appears to be PCORI's goal: to structure and generate that kind of evidence “to inform patients and clinicians about the different options and what the outcomes of those options are,” she said.

Ardis Dee Hoven, MD

Since PCORI's inception, the American Medical Association has called for a national, sustainable research infrastructure that involves practicing physicians and their organizations, Ardis Dee Hoven, MD, an infectious disease specialist and the AMA's president-elect, said during the forum. “Together we can support the development of a best-in-class research infrastructure that helps provide high-quality and up-to-date clinical information to aid in decision-making at the point of care,” she said.

The benefit to physicians

As discussions continue about establishing a network of networks, the new initiative offers a great opportunity for PCORI to collaborate with clinical registries, which “play a key role in priority-setting, dissemination and adoption” of comparative effectiveness research findings, Dr. Hoven said. Such registries offer an attractive option for PCORI, given their ability to incorporate these types of findings into clinical practice guidelines, and to adapt those findings into real-time practice on a widespread basis, she said.

PCORI's funding opportunity would give medical specialty societies additional resources to improve and expand upon use of their registries. The hope is that these societies will partner to participate in these networks, Dr. Hoven said.

Interested applicants must submit letters of intent by June 19. The applications are due Sept. 27, and awards will be announced in December. Several medical specialty societies that are involved with clinical registries said they had not determined their possible roles in the project.

Dr. Hoven stressed that PCORI's activities should not be limited to academic medical centers or other large health systems. Growing numbers of physicians have developed expertise with clinical registries as well as the use and adoption of electronic health records. “Leveraging this knowledge base represents an invaluable opportunity for PCORI,” she said. In the meantime, medical schools see the new data infrastructure as an opportunity “where residents, medical students and future researchers can work in an environment where electronic health records are shared, where there are new partnerships beyond the walls of academic medicine,” said Ann Bonham, PhD, chief scientific officer of the Assn. of American Medical Colleges.

Teaching hospitals and medical schools that care for the most vulnerable patients aren't just looking for opportunities to build up more information on claims data, but also to generate health statistics that point to inequities in health care, Bonham said.

Patient cooperation a crucial element

Participants at the forum discussed the importance of obtaining patient trust in developing this type of network. Many patients are hesitant about publicly disseminating their health information, said Nancy Davenport-Ennis, founder and CEO of the National Patient Advocate Foundation. She recalled a recent health care meeting in Washington during which a question was asked of the audience: How many of them would be comfortable with having their health information put into a national program to advance clinical research, if it were identifiable? “There were about 500 people in the audience, and only two raised their hands. So we've got to get past that point and help consumers understand how there would be an assuredness of privacy and security,” she said.

In discussing the idea of putting potentially identifiable information into public domains to promote the expansion of clinical research, Davenport-Ennis observed that it often was the sickest patients who were most likely to say, “Take whatever data you need, put it out there, just help us find answers.”

Bonham said the AAMC appreciated PCORI's goal of balancing the openness of data with the protection of human subjects and their privacy. “The first step is developing a sense of trust with those patients, particularly those vulnerable patients.”

ADDITIONAL INFORMATION

Elements of a patient-centered data infrastructure

The Patient-Centered Outcomes Research Institute, created by the Affordable Care Act to conduct comparative effectiveness research, is committing nearly $70 million to develop a national group of networks to advance this research. PCORI has outlined what characteristics such a framework ideally should include:

Possible participants in “network of networks”

The Patient-Centered Outcomes Research Institute is encouraging various sectors of health care to participate in, partner with or share data sources in developing a new patient-centered clinical data research network. Applications are due Sept. 27, and awards will be announced in December.

Organizations, or combinations of entities, that might be eligible for clinical data research network funding:

Two integrated health care delivery systems.

A health care delivery system and one or more health plans.

A health plan and two or more delivery systems.

A practice-based research network and a health plan.

An accountable care organization and its affiliates.

Organizations that might be eligible for patient-powered research networks funding:

A U.S.-based network, group or organization of patients of any size that has as a central goal the establishment and growth of an activated cadre of individuals who would provide patient-reported data for patient-centered outcomes research.

Networks or groups that have been developed in part through the efforts of clinicians, researchers or delivery systems to participate in comparative effectiveness research, including randomized clinical trials.

Networks or groups of patients who have been convened through the efforts of Internet-based or social media-based vendors, such as online communities, groups convened to use personal health records or groups formed specifically to participate in research.

Existing patient registries in which member patients are active in governance of registry activities or which aim to enlist and activate patients in the initial phase of this project.